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ARTICLE | Regulation

June 18 Quick Takes: Orphazyme looks ahead to EMA decision following complete response letter; plus Chi-Med, BeiGene, Blueprint and Agenus

June 18, 2021 11:04 PM UTC

Orphazyme A/S (NASDAQ:ORPH; CSE:ORPHA) is looking ahead to a CHMP opinion in 4Q21 for arimoclomol after receiving a complete response letter from FDA for the inducer of Hsp70 to treat Niemann-Pick disease type C (NPC). According to the company, FDA requested additional evidence to substantiate the validity and interpretation of the 5-domain NPC Clinical Severity Scale (NPCCSS), the primary endpoint in its Phase II/III study, as well as additional data. The company shed $7.23 (50%) to $7.33 on NASDAQ Friday. 

China’s National Medical Products Administration (NMPA) approved Sulanda surufatinib from  Hutchmed China Ltd. (NASDAQ:HCM; LSE:HCM) to treat pancreatic neuroendocrine tumors. The FGFR, VEGFR and CSF1R inhibitor, which was first approved by NMPA in December for non-pancreatic neuroendocrine tumors, is under review in the U.S...

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